Aplastic Anemia Market

• The United States accounted for the highest Aplastic Anemia Treatment Market Size, with nearly 70% of the Aplastic Anemia Market Share in the 7MM, as compared to EU4 and the UK and Japan in 2023.
• Aplastic Anemia is a rare and severe non-malignant disease characterized by autoimmune destruction of early hematopoietic cells. Its global Aplastic Anemia Incidence Rate ranges from 0.7 to 7.4 cases per million inhabitants per year, with higher rates in Asia compared to Europe and the United States.
• Based on severity, Severe and Very Severe Aplastic Anemia Patients contributes roughly 65%-80% of the total Aplastic Anemia patient population. 
• HSCT remains the standard of care in patients aged <40 years however IST remains the standard of care in patients >40 years and above. Severity is another important factor when deciding the most optimal therapy for Aplastic Anemia patients.
• Younger aplastic anemia patients experience a very good long-term survival, while that of patients ≥60 years in particular remains poor. Thus, an unmet need exists to improve the management of older aplastic anemia patients.
• Concerning pharmacological interventions, currently, Novartis' PROMACTA/REVOLADE is contributing the most in driving revenue followed by immunosuppressive therapies (IST) in the 7MM. As per Novartis, major drivers for PROMACTA/REVOLADE’s revenue are both Chronic Immune thrombocytopenic purpura (ITP) and Severe Apalastic Anemia Patients.
• Overall, even after the launch of emerging therapies and the recent approvals, the Aplastic Anemia Market Size is expected to be majorly driven by Immunosuppressive therapies (ISTs), and Promacta/Revolade .
• Key Aplastic Anemia Companies such as Regeneron Pharmaceuticals, are working on REGN7257 for refractory or relapsed SAA after immunosuppressive therapy, and Gamida Cell is developing umbilical cord blood-derived stem and progenitor cells to enhance engraftment and improve transplant outcomes in unrelated umbilical cord blood transplantation for SAA.
• Incorporating androgens and potentially other growth factors like G-CSF and erythropoietin alongside the current three-drug regimen shows potential for enhancing marrow function and facilitating the recovery of blood counts.
• Despite the advancements in treating aplastic anemia, there remain challenges in finding effective and well-tolerated treatments, especially for patients who do not respond to standard therapies or for those who may not be suitable candidates for more aggressive interventions
• Among the 7MM, Japan accounted for the highest total incident cases of Aplastic Anemia, contributing approximately 30% in 2023.

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Key Factors Driving the Aplastic Anemia Market

Rising Aplastic Anemia Incidence 

Aplastic anemia can occur in patients of any ethnicity, but according to many sources, it has an incidence rate of about 1–2 cases per million cases in the western regions of the US and Europe. As per DelveInsight estimates, the total Incident population of aplastic anemia in the 7MM were around 2,500 cases in 2024. These cases are projected to increase during the forecast period. 

Increase in PROMACTA‘s Patient Uptake

Novartis' PROMACTA/REVOLADE is contributing the most in driving revenue. PROMACTA‘s increase in patient uptake since 2018 (first line approval) and price hike of roughly 7% year on year since 2020 are contributing strongly to drive its market.

Launch of Emerging Aplastic Anemia Drugs

Some of the aplastic anemia drugs in clinical trials include REGN7257 (Regeneron Pharmaceuticals), OMISIRGE (omidubicel)(Gamida Cell), CK0801 (Cellenkos), and others.

Aplastic Anemia Therapeutics Market Report Summary 

• The detailed Aplastic Anemia Drugs Market report provides significant knowledge about epidemiological segments, including the historical and forecasted patient pool data, thus providing a thorough picture of anticipated future development in incident pool and treatment guidelines. It gives deep insights into various areas, allowing for a complete examination of the subject.
• The Aplastic Anemia Drugs Market report also includes an all-inclusive account of current management techniques and emerging therapies and elaborative profiles of late-stage (Phase III and Phase II) and prominent therapies that would impact the current treatment landscape and result in an overall market shift. The therapy profiles include a detailed assessment of the current and emerging therapies, information regarding the approval-based and ongoing trials, and a thorough drug description.
• The Aplastic Anemia Drugs Market report also encompasses a comprehensive analysis of the Aplastic Anemia market, providing an in-depth examination of its historical and projected Aplastic Anemia Treatment Market Size (2020–2034). It also includes the market share of therapies, detailed assumptions, and the underlying rationale for our methodology. The report also includes drug outreach coverage in the 7MM region.
• The Aplastic Anemia Drugs Market report includes qualitative as well as quantitative insights that provide an edge while developing business strategies by understanding trends through SWOT analysis and expert insights/KOL views, including experts from various hospitals and prominent universities, patient journey, which gives an overview of the patient landscape commencing from detection of the disease to treatment being administered to the patients, with the treatment preferences that help shape and drive the 7MM Aplastic anemia drugs market.

The table given below further depicts the key segments provided in the report:

Aplastic Anemia Therapeutics Market

Various key Aplastic Anemia Companies are currently investigating their Aplastic Anemia drug, such as Novartis, Pfizer, Teva Pharmaceuticals, Kyowa Kirin, Sanofi and others. The details of the country and therapy-wise market size have been provided below.

• The total Aplastic Anemia Treatment Market Size was found to be ~USD 270 million in 2023 in the 7MM, which is further expected to increase by 2034.
• Among the 7MM, Japan accounts for the second highest total aplastic anemia drugs market. The Aplastic Anemia Treatment Market Size in Japan has been assessed to be ~USD 35 million in 2023, expected to increase during the forcast period.
• During the forecast period, Aplastic Anemia pipeline candidates such as Omidubicel-onlv, REGN7257, and ALVAIZ (eltrombopag choline) are expected to drive the rise in Aplastic anemia market size.
• Among EU4 and the UK, Germany accounts for the highest Aplastic Anemia Treatment Market Size with ~USD 14 million in 2023, followed by France.
• Aplastic Anemia Treatment Market Size is expected to increase due to several factors such as an increase in disease incidence in Asian countries, high patient uptake of PROMACTA (until expected patent expiry in 2025), approval of Teva Pharma's ALVAIZ in the US, and Kyowa Kirin's ROMIPLATE in Japan along with the expected launch of potential emerging therapies

Aplastic Anemia Recent Developments

• In Aug 2025, Ayrmid Ltd. announced FDA acceptance of the priority review for omidubicel to treat Severe Aplastic Anemia, with a PDUFA date set for December 10, 2025, based on promising NIH study results.
• In April 2025, Cellenkos® Inc. announced that the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation to its novel T regulatory (Treg) cell therapy product, CK0801, aimed at treating inflammatory diseases and autoimmune disorders.

Aplastic Anemia Drugs Market Chapters

The Aplastic anemia drugs market report’s drugs section includes an in-depth examination of marketed drugs and late-stage pipeline therapeutics (Phase III and Phase II) for Aplastic anemia. The drug chapters section contains useful information on various aspects of Aplastic anemia clinical trials, including specific details such as the pharmacological mechanisms of the drugs involved, designations, approval status, patent information, and a comprehensive analysis of the pros and cons associated with each drug. It also includes the most recent Aplastic Anemia news updates and press releases on drugs that treat Aplastic Anemia.

Aplastic Anemia Marketed Therapies 

• ALVAIZ (eltrombopag choline): Teva Pharmaceuticals 

ALVAIZ (eltrombopag choline) is an oral thrombopoietin (TPO) receptor agonist that works to increase platelet production by interacting with the transmembrane domain of the human TPO-receptor to initiate signaling cascades that induce proliferation and differentiation of megakaryocytes. Lowest dose of ALVAIZ is use to achieve and maintain a hematologic response. Dose adjustments are based upon the platelet count. Hematologic response requires dose titration, generally up to 108 mg, and may take up to 16 weeks after starting ALVAIZ.

• ROMIPLATE (romiplostim; AMG531): Kyowa Kirin/Amgen

ROMIPLATE is composed of recombinant protein acting on the thrombopoietin receptor, which has been licensed from Amgen (K-A) to Kyowa Kirin. It was launched as a drug for idiopathic thrombocytopenic purpura (ITP) in April 2011 and for aplastic anemia in patients who had an inadequate response to conventional therapy in June 2019 in Japan. The drug is composed of recombinant protein stimulating hematopoiesis via acting on the thrombopoietin receptors; Acts directly on megakaryocytic progenitor cells and exerts platelet hematopoietic effects. In addition, this drug promotes the proliferation and differentiation of megakaryocytic progenitor cells by binding to and activating thrombopoietin receptors.

Aplastic Anemia Emerging Therapies

• REGN7257: Regeneron Pharmaceuticals

It’s a gc cytokine receptor antibody that targets the common g chain (gc; IL-2RG) found in interleukin (IL) receptors for various gc cytokines, including IL-2, IL-4, IL-7, IL-9, IL-15, and IL-21. It was developed to investigate the potential of targeting gc cytokines for the prevention and treatment of T-cell-mediated diseases. By blocking the signaling of GC cytokines, REGN7257 aims to ameliorate T-cell-mediated pathogenesis. This antibody offers a promising approach to understand better the role of gc cytokines in inflammatory and autoimmune disease settings, where the lack of appropriate neutralizing antibodies recognizing IL-2RG has been a challenge.

REGN7257 has the potential to provide valuable insights into disease mechanisms and may open up new therapeutic strategies for T-cell-mediated conditions. The drug REGN7257 is presently undergoing evaluation in a Phase I/II clinical trial to assess its safety and tolerability in patients with severe aplastic anemia (SAA) who have either relapsed after immunosuppressive therapy (IST) or are refractory to IST.

• OMISIRGE (omidubicel): Gamida Cell

OMISIRGE is a nicotinamide-modified allogeneic hematopoietic progenitor cell therapy derived from cord blood. It is indicated for use in adults and pediatric patients aged 12 years and older with hematologic malignancies who are planned for umbilical cord blood transplantation following myeloablative conditioning. The therapy aims to reduce the time to neutrophil recovery and the incidence of infection in this patient population. Omisirge has received approval for use in hematological malignancies and is currently undergoing evaluation in an ongoing investigator-sponsored Phase I/II study focusing on patients with severe aplastic anemia.

Note: Detailed assessment will be provided in the final report of Aplastic anemia……

Aplastic Anemia Market Outlook

The Aplastic Anemia Prognosis, varies based on the severity of the condition. Some cases can be successfully treated, while others may progress to severe complications, such as myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML).

Historically, the primary modalities for treating aplastic anemia have been IST and BMT in eligible patients. For individuals with SAA/VSAA deemed suitable for transplant-based interventions, age emerges as a pivotal determinant influencing survival post-matched sibling donor (MSD) allogeneic transplantation. Notably, older patients, often having undergone prior IST, presenting with additional comorbidities, diminished performance status, and an extended duration between diagnosis and BMT, exhibit distinctive challenges in outcomes, with fludarabine-containing regimens offering more promising results in this demographic, albeit based on limited retrospective analyses.

In real-world scenarios, the approach to treating NSAA differs significantly from SAA/VSAA. Unlike SAA/VSAA, hematopoietic stem cell transplantation (HSCT) is not employed in NSAA cases. The criteria for determining when to initiate treatment for NSAA are less clearly defined, and there is a lack of comprehensive recommendations for clinical evaluation and therapeutic strategies. The ultimate management of NSAA predominantly relies on the expertise of individual medical centers.

Since, some potential therapies are being investigated to manage aplastic anemia, predicting that the treatment space will experience significant reconstitution during 2024–2034 is safe.

Aplastic Anemia Treatment Market: Overview

Aplastic anemia is a rare and severe nonmalignant disease characterized by autoimmune destruction of early hematopoietic cells. Aplastic anemia can be categorized into congenital and acquired forms. Inherited forms are rare and include Fanconi Anemia, Congenital Keratosis, Congenital Pure Red Cell Aplasia, and Shwachman–Diamond Syndrome. Most cases of acquired aplastic anemia occur unrelated to any identifiable causes or for unknown reasons. Researchers believe that most are due to the immune system mistakenly targeting the bone marrow. Specific symptoms vary from case to case. Some individuals may have mild symptoms that remain stable for many years; others may have serious symptoms that can progress to life-threatening complications.

Further details are provided in the report…

Aplastic Anemia Diagnosis

Initial screening evaluation of a patient with aplastic anemia is required to document pancytopenia with a hypocellular marrow, followed by testing to exclude alternative diagnoses. Aplastic anemia severity and outcomes of a transplant evaluation factor into determining an optimal treatment strategy. Patients with severe or very severe aplastic anemia (SAA/VSAA) 40 years of age or younger with an HLA-matched sibling donor should undergo an evaluation for an allogeneic bone marrow transplant; older patients or patients without an HLA-identical sibling donor should be evaluated for frontline immunosuppressive therapy. Patient age, family history, exposure to toxic substances or medicaments, infections, as well as occupation, clinical presentation, and comorbidities are relevant information that helps to set priorities in the diagnostic process. 

Further details related to country-based variations are provided in the report…

Aplastic Anemia Treatment

Bone marrow transplantation, specifically an allogeneic transplant, is the treatment of choice in children and younger adults. With an allogeneic bone marrow transplant, an affected individual’s abnormal bone marrow cells are eradicated or destroyed by chemotherapy and replaced with healthy marrow obtained from a donor. The donor marrow is transplanted by injecting the cells of the donor intravenously into the patient’s body, where it travels to the patient’s bone marrow and eventually begins producing new blood cells. The best match for a bone marrow transplant is an identical twin, sibling, or close relative who shares most of the same genetic makeup as the patient.

Aplastic Anemia Epidemiology

The Aplastic Anemia epidemiology chapter in the report provides historical as well as forecasted epidemiology segmented by Aplastic Anemia Incidence cases, Aplastic Anemia severity-specific cases, and Aplastic Anemia age-specific cases, in the United States, EU4 countries (Germany, France, Italy, Spain), and the United Kingdom, and Japan from 2020 to 2034. 

• In 2023, approximately 56% of aplastic anemia cases were reported in individuals aged 60 and above, while the remaining 43% were below the age of 60 in the 7MM.
• Among the EU4 countries, the highest aplastic anemia incidence cases were estimated to be in Germany accounting for around .
• In 2023, the incident cases of non-severe aplastic anemia, severe aplastic anemia, and very severe aplastic anemia in the United States were ~100, ~400, and ~200.
• Due to the higher disease burden in Asian countries compared to the West and historical growth rates from recent epidemiological studies, Aplastic Anemia patients are anticipated to increase in Japan by 2034.

KOL Views

In order to stay abreast of the latest trends in the Aplastic Anemia Treatment Market, we conduct primary research by seeking the opinions of Key Opinion Leaders (KOLs) and Subject Matter Experts (SMEs) who work in the relevant field. This helps us fill any gaps in data and validate our secondary research. We have reached out to industry experts to gather insights on various aspects of Aplastic Anemia, including the evolving Aplastic Anemia Treatment Market Landscape, patients’ reliance on conventional therapies, their acceptance of therapy switching, drug uptake, and challenges related to accessibility. The experts included medical/scientific writers, professors, and researchers from prestigious universities in the US, Europe, the UK, and Japan.

Our team of analysts at DelveInsight connected with more than 10 KOLs across the 7 Major Markets (7MM). We contacted institutions such as Stanford Medicine, University of Barcelona and others. By obtaining the opinions of these experts, we gained a better understanding of the current and emerging treatment patterns in the Aplastic Anemia market, which will assist our clients in analyzing the overall epidemiology and market scenario. 

The opinions of experts from various regions have been provided below: 

Aplastic Anemia Treatment Market: Qualitative Analysis

We conduct qualitative and Aplastic Anemia drugs market intelligence analysis employing various methods, including SWOT Analysis and Conjoint Analysis. Strengths, weaknesses, opportunities, and threats in disease diagnosis, patient awareness, patient burden, competitive landscape, cost-effectiveness, and geographical accessibility of therapies are described in the SWOT analysis. These recommendations are based on the Analyst’s evaluation of the patient burden, cost analysis, and the current and emerging therapy landscape. Conjoint Analysis compares the effectiveness and safety of numerous approved and emergent drugs depending on key criteria such as frequency of administration, designation, route of administration, and order of entry. To assess the success of therapy, several factors such as overall response rate, complete response, partial response, etc. have been evaluated.

Furthermore, the drug’s safety is analyzed, in which acceptability, tolerability, and adverse events are closely monitored, and it establishes a firm grasp of the side effects of the drugs used in the trials. Furthermore, for each therapy, the rating is based on the route of administration, sequence of entrance and designation, chance of success, and addressable patient pool. These characteristics determine the ultimate weightage score and ranking of developing therapeutics. 

Aplastic Anemia Treatment Market Access and Reimbursement

Because newly authorized Aplastic Anemia drugs are often expensive, some patients escape receiving proper treatment or use off-label, less expensive prescriptions. Reimbursement plays a critical role in how innovative treatments can enter the market. The cost of the medicine, compared to the benefit it provides to patients who are being treated, sometimes determines whether or not it will be reimbursed. Regulatory status, target population size, the setting of treatment, unmet needs, the number of incremental benefit claims, and prices can all affect market access and reimbursement possibilities. 

The Aplastic Anemia drugs market report further provides detailed insights on the country-wise accessibility and reimbursement scenarios, cost-effectiveness scenario of approved therapies, programs making accessibility easier and out-of-pocket costs more affordable, insights on patients insured under federal or state government prescription drug programs, etc.

Aplastic Anemia Treatment Market Report Insights

• Patient-based Aplastic Anemia Market Forecasting
• Therapeutic Approaches
• Aplastic Anemia Treatment Market Size of each therapy
• Aplastic Anemia Treatment Market Size
• Aplastic Anemia Market Trends
• Existing Aplastic Anemia drugs market Opportunity 

Aplastic Anemia Treatment Market Report Key Strengths

• 11 Years Aplastic Anemia Market Forecast
• The 7MM Coverage 
• Aplastic Anemia Epidemiology Segmentation (Segmented by incident cases, type, and gender-specific Cases)
• Key Cross Competition evaluating the marketed as well as emerging therapies

Aplastic Anemia Treatment Market Report Assessment

• Current Aplastic Anemia Treatment Market and Management Practices
• Aplastic Anemia Unmet Needs
• Aplastic Anemia drugs market Attractiveness
• Qualitative Analysis (SWOT, Conjoint Analysis)

Key Questions

• Would there be any changes observed in the current Aplastic Anemia treatment approach?
• Will there be any improvements in Aplastic Anemia management recommendations?
• Would research and development advances pave the way for future tests and Aplastic Anemia therapies?
• Would the innovations in diagnostic tests of Aplastic Anemia space experience a significant impact and lead to a positive shift in the treatment landscape of Aplastic Anemia?
• What kind of uptake will the new therapies witness in the coming years in Aplastic Anemia patients?

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